ArticleMolecular therapy. Advances2026
Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Adeno-associated virus (AAV) vector-based liver gene therapy for inherited diseases has demonstrated efficacy in clinical trials in adults. However, its application to pediatric patients is limited by loss of AAV genomes during hepatocyte proliferation, compromising long-term benefits. Additionally, the anti-AAV immune responses induced after the initial AAV-administration preclude vector re-dosing. One key driver of this immune response is mTOR-dependent activation of dendritic cells. Inhibition of this pathway with rapamycin can promote immune tolerance. We assessed the safety and efficacy of rapamycin-loaded synthetic nanoparticles (ImmTOR) in juvenile OTC
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